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Term of the Day

Natural history study

A natural history study is a preplanned observational study intended to track the course of a disease over time, identifying demographic, genetic, environmental and other variables that correlate with its development and outcomes in the absence of intervention, or under standard of care. Designs may be retrospective (chart review of existing records) or prospective (longitudinal follow-up of a cohort or registry).

Natural history data is particularly important in rare and paediatric diseases, where randomised placebo-controlled trials may be infeasible or unethical. The FDA (guidance on rare disease natural history studies, 2019) and the EMA accept well-designed natural history studies to define endpoints and biomarkers, identify patient subgroups, estimate sample sizes and, in some cases, serve as external or historical control arms for single-arm trials supporting orphan products.

Because they are non-interventional, natural history studies fall outside the CTR and are governed by national law (for example France's MR-003 or MR-004 reference methodologies) and by the GDPR. They typically involve secondary use of medical records, long-term follow-up, genetic data and small populations in which anonymisation is rarely achievable, so pseudonymisation, a DPIA and a robust research legal basis under Art. 9(2)(j) are essential. Registries maintained by patient organisations or academic consortia raise additional questions of joint controllership and data access governance.

I

Investigational New Drug (IND) application

An Investigational New Drug (IND) application is the regulatory submission to the US Food and Drug Administration under 21 CFR Part 312 that a sponsor must file before shipping an unapproved drug across state lines and administering it to humans in a clinical trial. It is the US counterpart of the EU clinical trial application under the CTR.

An IND contains animal pharmacology and toxicology data, manufacturing information (CMC), clinical protocols, investigator information and the investigator's brochure. The FDA has 30 days to review the initial submission; if it does not impose a clinical hold, the trial may proceed. The IND remains open throughout development, with protocol amendments, safety reports, and annual reports submitted against it. Commercial INDs are distinguished from research INDs and from expanded-access INDs for treatment use.

For European sponsors, an IND brings EU trial data within the FDA's reach: individual patient data in SDTM and ADaM format, safety reports and, during Bioresearch Monitoring inspections, source documents at EU sites. These disclosures to a US federal agency are international transfers under the GDPR without an adequacy decision, generally justified under Art. 49(1)(d), and must be disclosed to participants in the informed consent form. Conversely, US sites enrolled under an IND in an EU-sponsored trial operate under HIPAA and the Common Rule, requiring a HIPAA authorisation in the consent package.

Does an IND allow a sponsor to market an investigational drug?

No. An IND supports investigational use under the applicable US requirements; it is not marketing approval. The submission provides information on areas such as manufacturing, non-clinical safety and the proposed clinical investigation. Keep the investigational pathway separate from the later evidence and application needed to seek commercial authorisation.